Gene therapy phase II trial shows promise for severe heart failure

Researchers at Mount Sinai Medical Center have developed a gene therapy for patients with heart failure.

In a phase II clinical trial, patients with severe heart failure either improved or remained stable after receiving high dose gene therapy called SERCA2a.

The results, published in the American Heart Association journal Circulation, showed the gene therapy, which is delivered by an inert virus into cardiac cells, improved pumping action of the heart by stimulating an enzyme.

Improvement or stabilization was seen in advanced heart failure patients given the gene therapy, after one year.

Roger J. Hajjar, MD, Research Director of Mount Sinai's Wiener Family Cardiovascular Research Laboratories, and the Arthur and Janet C. Ross Professor of Medicine, and Gene and Cell Medicine, Mount Sinai School of Medicine said, “Few treatment options have shown such improved clinical outcomes in this patient population in the last decade.”

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The gene therapy “establishes a new paradigm” in advanced heart failure treatment, explains Hajjar, because it validates the effectiveness of SERCA2a. It also shows viruses can safely be used to deliver gene therapy.

The study included 39 patients, randomly chosen to receive SERCA2a or placebo in the CUPID (Calcium Up-regulation by Percutaneous administration of gene therapy In cardiac Disease) trial.

The gene therapy is administered during cardiac catheterization, directly into the heart.

The patients were followed for a year. In addition to improved heart function, the patients receiving the therapy had .4 days of hospitalization, compared to those given placebo whose average admission stay was 4.5 days.

Dr. Hajjar, who was one of the researchers in the study, has an equity interest in Celladon Corporation who funded the study, and participates on an Advisory Board. The phase II trial shows gene therapy with SERCA2a could provide another treatment option for patients living with congestive heart failure.